EP4206213A1 — Compounds and methods for modulation of smn2
Assigned to Ionis Pharmaceuticals Inc · Expires 2023-07-05 · 3y expired
What this patent protects
Disclosed herein are compounds, compositions and compounds for use in modulating splicing of SMN2, said compounds consisting in oligomers comprising a modified oligonucleotide consisting of 14-25 linked nucleosides, wherein the modified oligonucleotide is complementary to an SMN2…
USPTO Abstract
Disclosed herein are compounds, compositions and compounds for use in modulating splicing of SMN2, said compounds consisting in oligomers comprising a modified oligonucleotide consisting of 14-25 linked nucleosides, wherein the modified oligonucleotide is complementary to an SMN2 pre-mRNA; and wherein at least one nucleoside of the modified oligonucleotide comprises a 2'-O(N-alkyl acetamide) modified sugar moiety. Also provided are uses of disclosed compounds and compositions in the manufacture of a medicament for treatment of spinal muscular atrophy.
Drugs covered by this patent
Bibliographic data sourced from FDA Orange Book + USPTO public records. Plain-English summary generated by AI grounded in source text. Patent term extensions (PTR, SPC, pediatric) may shift the effective expiry. Not legal advice.
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